The approval of two gene therapies to treat sickle cell disease has given hope to patients suffering from the debilitating disease, which overwhelmingly affects blacks and people of color.
Health officials now face a challenge to find a way to provide equitable access to expensive treatments.
Crippling episodes of pain from the genetic blood disorder make life unpredictable for patients like Michael Goodwin. Sickle cell has forced him to quit his job and at times takes him away from his family.
“I can sometimes be in the hospital 20 days a month,” Goodwin, 36, said. “I’ve been to the hospital a lot more as I’ve gotten older, which hurts me because now I have a son and I’m married.”
But he is reluctant to try the new one-time gene therapies because they require months of intensive medical preparation, including chemotherapy, to prepare patients’ bone marrow stem cells for gene extraction and editing.
Goodwin also worries about the cost. Vertex PharmaceuticalsGene therapy Casgevy lists for $2.2 million, while Bluebird BioLyfgenia’s treatment reports for $3.1 million.
“I have insurance, but … I already have medical bills,” she said.
The treatments were hailed as a long-awaited breakthrough to treat the disease when they were approved in December. But barriers to ensuring equitable access and a lack of infrastructure to manage nascent treatments have raised questions about how many people will benefit from them.
Patient blood sample positive for sickle cell.
Kitsawet Saethao | Istock | Getty Images
Goodwin’s reluctance to seek treatment comes as no surprise to Dr. Julie Kanter, director of the Adult Sickle Clinic at the University of Alabama at Birmingham.
“My guess is that even if we opened the gates today to everyone who gets this treatment, at most only 10 percent of those people with sickle cell disease would want this treatment,” said Kanter, who serves also as president of the National Alliance of Sickle. Cell Centers. “And even that would be too much to handle right this second.”
More than 100,000 Americans have sickle cell disease, according to estimates from the Centers for Disease Control and Prevention, and between 50% and 60% of those who are covered are covered by the federal and state Medicaid insurance program.
Kanter said it will take time to build capacity and build facilities across the country to treat patients at scale.
“We’re really hoping that having the National Alliance of Sickle Cell Centers will allow us to empower our centers to generally better care for people living with this disease, which we haven’t been able to do in the past because cost is an issue,” he said. he said.
High costs bring a new payment model
As they figure out how to increase treatment capacity, state and federal officials are grappling with how to provide access to the costly new treatments for thousands of patients covered by the Medicaid safety net program.
“It gives us an opportunity to respond to people with medical conditions for which there haven’t been very satisfactory treatments. But I think the immediate issue is that the costs are very high. And government budgets just can’t handle it on their own,” Kate said. McEvoy, executive director of the National Association of Medicaid Directors.
University of Washington analysis found that at a price of $2 million or less, single gene therapy treatments would provide an acceptable value, offsetting medical and lifestyle costs for patients with acute sickle cell disease. Many with the disease require multiple hospitalizations and blood transfusions, which can leave them unable to work.
But the researchers concluded that a lower price tag closer to $1 million would help ensure greater access.
The Biden administration is starting negotiations in the coming weeks with Vertex and Bluebird Bio to get rebates for state Medicaid programs, with payments tied to patient health outcomes. It is part of the Centers for Medicare & Medicaid Services’ Model of access to cell and gene therapy, which aims to make it easier to obtain high-priced new treatments. The approval of the sickle cell therapies prompted the administration to start implementing the new payment demonstration program a year earlier, starting in January 2025.
“There are probably about 100 treatments in the pipeline at the FDA at an advanced stage of application … so this is a real-time priority in terms of developing strategies that will degrade the ability of Medicaid programs to cover the treatments,” McEvoy said.
Direct talks with sickle cell drug makers are the way big pharma wants them Merck, Eli Lilly and others are suing the Biden administration over the inflation-reducing Medicare price negotiations that began in February. Those talks could see sharply lower prices for the first 10 drugs selected for negotiation.
However, on Vertex’s quarterly earnings call this month, executives expressed confidence in the negotiation process in this case. They said discussions with individual state Medicaid agencies will help ensure broad access and address long-standing care disparities in the sickle cell community.
“We are not waiting for the demonstration before we secure access for patients covered by Medicaid,” Steve Arbuckle, executive vice president and CEO of Vertex, told analysts. “If you look at the profile of Casgevy, it’s so incredibly strong that we’re really talking about an outcome-based agreement that’s looking at whether a very, very small number of patients might not respond.”
Employers take note
Private employer health plans are also grappling with how to pay for a growing number of innovative treatments with seven-figure prices, said Morgan Health CEO Dan Mendelson, whose company focuses on workplace health programs.
“A lot of employers are looking at cell and gene therapies, looking at the costs, and subtracting them from their benefits. They know the therapies are valuable,” Mendelson said. But for smaller companies, “one case could exceed the cost of insuring an entire population over the course of a year, and the employer doesn’t even know if the employee will be left behind.”
Morgan Health is exploring new risk-sharing payment models that could help small and medium-sized businesses cover the rising costs of specialty treatments on the market.
Goodwin is covered by his wife’s employer’s health insurance. He hasn’t explored what kind of coverage her plan will provide for the new sickle cell treatments, because he’s still not sure if they’re right for him.
“If they could guarantee me the result – that I wouldn’t have sickle cell … I would do it in a heartbeat. In a heartbeat,” she said.
In addition to discussions about payments, Vertex and Bluebird Bio are taking steps to educate physicians and patient communities about the benefits of their new treatments.
Vertex expects its first commercial patient to begin treatment in the coming weeks. Bluebird said it expected its first patient to start Lyfgenia in the first quarter.